A Study to Investigate the Efficacy, Safety, and Tolerability of JNJ-42165279 in Adolescent and Adult Participants With Autism Spectrum Disorder

NCT03664232 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: CR10827542165279AUT2001
• Study Type: interventional
• Target: 78
• Locations: 1 country
• Sites: 9

Brief Summary

The purpose of this study is to evaluate the efficacy of JNJ-42165279 compared with placebo in the improvement of symptoms of Autism Spectrum Disorder (ASD) during 12 weeks of treatment using the Autism Behavior Inventory (ABI).

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (4)

• Change From Baseline to Day 85 in the Autism Behavior Inventory (ABI) Core Domain Score (Social Communication and Restrictive Behaviour)

  ABI:62-item questionnaire to track outcomes in autism spectrum disorder (ASD). Each item was answered on 1 of 2 possible dimensions: quality (how well person carries out particular behavior; 1 to 13 items) or frequency (how often particular behavior occurs; 14 to 62 items). Each item was rated on scale of 0 (never) to 3 (very often) for frequency and 0 (without help) to 3 (not at all) for quality. Higher score=severe symptoms/more frequency. ABI core domain score was from 2 domains: social communication (23 items; 3 sub-domains) and repetitive/restrictive behavior (RRB;15 items; 4 sub-domains). Domain and sub-domain scores: calculated as average of non-missing items (ie, sum of all non-missing items divided by number of non-missing items); scores ranged from 0 to 3, higher scores=more severe symptoms of ASD. ABI core domain score=sum of social communication and RRB domain scores divided by total number of items in these 2 domains. Negative changes in ABI core domain score=improvement.

  Baseline (Day 1) to Day 85

• Change From Baseline to Day 85 in the ABI Social Communication Domain Score

  ABI was 62-item questionnaire completed on a web/mobile application or on paper. It tracks outcomes in ASD. Each ABI item was answered on 1 of 2 possible dimensions, quality (how well a person carries out a particular behavior; 1 to 13 items) or frequency (how often a particular behavior occurs; 14 to 62 items). Each item was rated on scale of 0 (never) to 3 (very often) for frequency and 0 (without help) to 3 (not at all) for quality. Higher score indicated severe symptoms/more frequency. The ABI social communication domain score consisted of 23 items; with 3 sub-domains. The domain and sub-domain scores were calculated as the average of the non-missing items (ie, sum of all non-missing items divided by the number of non-missing items). For both domains and its sub-domains, scores ranged from 0 to 3 with higher scores indicating more severe symptoms of ASD. Negative change in score indicates improvement.

  Baseline (Day 1) to Day 85

• Change From Baseline to Day 85 in the ABI Repetitive/Restrictive Behavior (RRB) Domain Score

  ABI is 62-item questionnaire completed on a web/mobile application or on paper. It tracks outcomes in ASD. Each ABI item was answered on 1 of 2 possible dimensions, quality (how well a person carries out a particular behavior; 1 to 13 items) or frequency (how often a particular behavior occurs; 14 to 62 items). Each item was rated on scale of 0 (never) to 3 (very often) for frequency and 0 (without help) to 3 (not at all) for quality. Higher score indicated severe symptoms/more frequency. The ABI RRB domain score consisted of 15 items; with 3 sub-domains. The domain and sub-domain scores were calculated as the average of the non-missing items (ie, sum of all non-missing items divided by the number of non-missing items). For both domains and its sub-domains, scores ranged from 0 to 3 with higher scores indicating more severe symptoms of ASD. Negative change in score indicates improvement.

  Baseline (Day 1) to Day 85

• Change From Baseline to Day 85 in the Social Responsiveness Scale 2 (SRS-2) Total T-Score

  SRS-2: 65-item scale measured extent of autistic social impairment and included 5 subscales: social awareness, social cognition, social communication, social motivation, and restricted interests and repetitive behavior. Each of 65 items had 4 responses: not true, sometimes true, often true, and almost always true. Scoring value for each item was 0 to 3. If a response to an item was missing, then pre-defined median value for item (0 or 1) was imputed. SRS-2 was not scored if 7 or more item responses were missed. Total raw score was sum of item response values. Each subscale was obtained by adding response values and converted total raw score to standardized T-score based on gender and rater (parent or caregiver). Total T-score was categorized: within normal limits (\<=59), mild (60 to 65), moderate (66 to 75) and severe (\>=76). For total score T-score, higher scores=more severe symptoms. SRS T-score had mean of 50 and standard deviation of 10. Negative changes in T-scores=improvement.

  Baseline (Day 1) to Day 85

Secondary Outcomes (16)

• Change From Baseline to Day 85 in the ABI Mood and Anxiety Domain Score

  Baseline (Day 1) to Day 85

• Change From Baseline to Day 85 in the ABI Challenging Behavior Domain Score

  Baseline (Day 1) to Day 85

• Change From Baseline to Day 85 in the ABI Self-Regulation Domain Score

  Baseline (Day 1) to Day 85

• Change From Baseline to Day 85 in the Aberrant Behavior Checklist (ABC) Subscales Scores

  Baseline (Day 1) to Day 85

• Change From Baseline to Day 71 in the Autism Behavior Inventory-Short Form (ABI-S) Domains Scores

  Baseline (Day 1) to Day 71

Study Arms (2)

JNJ-42165279

EXPERIMENTAL

Participants will self-administer 25 milligram (mg) JNJ-42165279 tablets orally twice daily for 12 weeks.

Drug: JNJ-42165279

Placebo

PLACEBO COMPARATOR

Participants will self-administer matching placebo tablets orally twice daily for 12 weeks.

Drug: Placebo

Interventions

JNJ-42165279 DRUG

Participants will receive 25 mg JNJ-42165279 orally twice daily for 12 weeks.

JNJ-42165279

Placebo DRUG

Participants will receive a matching placebo orally twice daily for 12 weeks.

Placebo

Eligibility Criteria

• Age: 13 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of Autism Spectrum Disorder (ASD) according to Diagnostic and Statistical Manual of Mental Disorders - 5th Edition (DSM-5) criteria and made or confirmed using the Autism Diagnostic Observation Schedule, 2nd edition (ADOS-2) (minimum score of 8 \[autism spectrum\])
• Otherwise healthy for their age group or medically stable with or without medication on the basis of physical examination, medical history, vital signs, 12-lead electrocardiogram (ECG), and clinical laboratory tests
• Have a composite score on Kaufman Brief Intelligence Test, Second Edition (KBIT-2) of at least 60
• Must live with a parent or primary caregiver or, if not, during each week he/she must either (A) spend at least 3 hours a day for at least 4 days or, (B) spend the weekend with a parent or primary caregiver
• Any pharmacologic, diet, or behavioral intervention for ASD must have begun at least 1 month prior to the baseline visit and continue unchanged through the treatment period, or have ended at least 1 month prior to the baseline visit
• Must be able to swallow the study medication whole and self-administer medication if living independently or have a parent or caregiver be able to administer medication
• Must agree to abide by the birth control requirements during the study and for 3 months after the last dose

You may not qualify if:

• Current or recent history of clinically significant suicidal ideation within the past 6 months, or a history of suicidal behavior within the past year
• Use of a drug with moderate/strong cytochrome P450 (CYP)3A4 inhibiting or inducing properties at, or prior to, screening that is not discontinued at least within 1 month prior to Day 1
• History of drug or alcohol use disorder according to DSM-5 criteria within 6 months before screening or positive test result(s) for alcohol or drugs of abuse (except if related to current treatment)
• Currently taking or has taken within the past month recreational or medically prescribed cannabis

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (9)

Harmonex Neuroscience Research

Dothan, Alabama, 36303, United States

Location

Southwest Autism Research and Resource Center

Phoenix, Arizona, 85006, United States

Location

NRC Research Institute

Orange, California, 92868, United States

Location

New York Presbyterian Hospital

New York, New York, 10032, United States

Location

Nathan Kline Institute

Orangeburg, New York, 10962, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Cutting Edge Research Group

Oklahoma City, Oklahoma, 73116, United States

Location

BioBehavioral Research of Austin PC

Austin, Texas, 78759, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

JNJ-42165279

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Study Director
• Organization: Janssen Research & Development, LLC

ClinicalTrialDisclosure@its.jnj.com

844-434-4210

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 7, 2018
• First Posted: September 10, 2018
• Study Start: November 7, 2018
• Primary Completion: October 17, 2022
• Study Completion: October 28, 2022
• Last Updated: April 23, 2025
• Results First Posted: April 23, 2025

Record last verified: 2025-04

Locations

US (9)